ArticleHaemophilia : the official journal of the World Federation of Hemophilia2023
Association of factor expression levels with annual bleeding rate in people with haemophilia B.
Article in Haemophilia : the official journal of the World Federation of Hemophilia, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed, 13 citations in OpenAlex.
- Safety, efficacy, and patient-reported outcomes 6 years after fidanacogene elaparvovec in adults with hemophilia B.Blood advances · 2026Trial
- Six-year FIX-padua gene therapy in hemophilia B.Blood advances · 2026Article
- Haemophilia B: an illustrative review of current challenges and opportunities.Research and practice in thrombosis and haemostasis · 2025Article
- The association of factor VIII activity levels with bleeding and quality of life in haemophilia a: findings from the European CHESS II study.Orphanet journal of rare diseases · 2025Article
- ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B.European journal of haematology · 2025Article
- Association of factor expression levels with annual bleeding rate in people with haemophilia B.Haemophilia : the official journal of the World Federation of Hemophilia · 2023Article
Corrections and comments
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Authors and funding
9 authors at 7 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionGene therapy clinical trials measure steady-state clotting factor expression levels (FELs) to evaluate the modulation of the bleeding phenotype, aiming to offer consistent protection against breakthrough bleeding events. The link between FELs and bleeding risk in people with haemophilia B (PwHB) is not well understood.
aimWe evaluated the association between FEL and ABR in PwHB.
methodsThis cross-sectional study extended the CHESS burden of illness studies in Europe and the United States. Recruitment of additional adult males with haemophilia B supplemented the existing CHESS sample size of PwHB and FELs. PwHB receiving prophylaxis were excluded, as fluctuating FELs may have confounded the analysis. Demographic and clinical characteristics were reported descriptively. Any recorded baseline FEL was reported by the haemophilia-treating physicians according to the medical records. Generalised linear models with log link explored the association between changes in FEL and ABR.
resultsThe study included 407 PwHB and no inhibitors receiving on-demand treatment. Mean age was 36.7 years; 56% from the EU, 44% from the United States. Mean baseline FEL was 9.95 IU/dl (SD, 10.47); mean ABR was 2.4 bleeds/year (SD, 2.64). After adjusting for covariates, the model showed that for every 1% increase in FEL the average ABR decreased by .08 (p < .001). Predicted number of bleeding events according to FEL showed a significant non-linear relationship between FEL and ABR (p < .05).
conclusionThis analysis showed a significant relationship between FEL and ABR, where increases in FEL were associated with decreases in ABR among men with HB in Europe and the US.
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